Core Insights - Ultragenyx Pharmaceutical reported a third-quarter 2025 loss of $1.81 per share, which was wider than the Zacks Consensus Estimate of a loss of $1.23, and compared to a loss of $1.40 per share in the same quarter last year [1][6] - Total revenues for the quarter were $159.9 million, reflecting a 15% year-over-year increase, but fell short of the Zacks Consensus Estimate of $168 million [1][6] Revenue Breakdown - Crysvita generated total revenues of $111.9 million, up 14% year over year, with contributions of $57 million from North America, $47 million from Latin America and Turkey, and $8 million from Europe [4] - Mepsevii product revenues declined by 27% year over year to $7 million, while Dojolvi revenues increased by 14% to $24.3 million due to new patient demand [5] - Evkeeza recorded sales of $16.7 million, up 57% as the drug continues to be launched in territories outside the United States [5][6] Operating Expenses - Operating expenses for the quarter were $330.8 million, a 22% increase year over year, driven by higher investments in late-stage pipeline programs and marketing costs [8] - Research and development expenses were $216.2 million (up 27%), selling, general and administrative expenses were $86.6 million (up 8%), and cost of sales was $28 million (up 33%) [8] Financial Guidance - The company expects total revenues in 2025 to be between $640 million and $670 million, representing growth of approximately 14-20% compared to 2024 [10] - Crysvita revenues are projected to be in the range of $460-$480 million (up 12-17% year over year), while Dojolvi revenues are expected between $90 million and $100 million (up 2-14% year over year) [10] Pipeline Updates - Ultragenyx faced a setback with the FDA issuing a complete response letter for its biologics license application for UX111, requesting more information related to manufacturing [12] - The company plans to resubmit the BLA for UX111 early in 2026 [13] - GTX-102 received Breakthrough Therapy designation from the FDA for treating Angelman syndrome, with data expected in the second half of 2026 [14] - The company has begun the rolling submission of a BLA for its investigational gene therapy DTX401, expected to be completed in Q4 2025 [15]

Core Insights - Ultragenyx Pharmaceutical reported a second-quarter 2025 loss of $1.17 per share, which is an improvement from a loss of $1.52 per share in the same quarter last year and better than the Zacks Consensus Estimate of a loss of $1.27 [1][5] - Total revenues for the quarter reached $166.5 million, reflecting a 13% year-over-year increase, driven primarily by higher product sales, and surpassing the Zacks Consensus Estimate of $162 million [1][5] Revenue Breakdown - Crysvita generated total revenues of $120.4 million, up 6% year over year, with contributions of $79 million from North America, $35 million from Latin America and Turkey, and $7 million from Europe [3] - Mepsevii product revenues increased by 35% year over year to $8.3 million, while Dojolvi revenues rose 20% to $23.2 million due to new patient demand [4] - Evkeeza recorded sales of $14.6 million in the first quarter, showing significant growth as the drug continues to be launched in territories outside the United States [4] Financial Guidance - The company reaffirmed its 2025 financial guidance, expecting total revenues between $640 million and $670 million, which represents a growth of approximately 14-20% compared to 2024 [9] - Crysvita revenues are anticipated to be in the range of $460-$480 million, reflecting a year-over-year increase of 12-17%, while Dojolvi revenues are expected to be between $90 million and $100 million, up 2-14% year over year [9] Operating Expenses - Operating expenses for the quarter were $274.4 million, a 4% increase year over year, attributed to higher investments in late-stage pipeline programs and marketing costs for approved drugs [7] - Research and development expenses were $164.7 million (up 2%), selling, general and administrative expenses were $86.6 million (up 7%), and cost of sales was $23 million (up 8%) [7] Pipeline Updates - The FDA issued a complete response letter for Ultragenyx's biologics license application for UX111, requesting additional information related to manufacturing processes, which the company plans to address promptly [11][12] - The company is also developing GTX-102 for Angelman syndrome, which received Breakthrough Therapy designation, with data expected in the second half of 2026 [14] - Ultragenyx plans to submit a BLA for DTX401, a gene therapy for glycogen storage disease type Ia, in the fourth quarter of 2025 [15]

Group 1 - Pomerantz LLP is investigating claims on behalf of investors of Mereo BioPharma Group plc regarding potential securities fraud or unlawful business practices by the company and its officers or directors [1] - On July 9, 2025, Mereo and Ultragenyx Pharmaceutical Inc. announced the progress of the Phase 3 Orbit study for UX143 (setrusumab) in patients with osteogenesis imperfecta, with a final analysis expected by the end of the year [2] - Following the announcement, Mereo's ADR price dropped by $1.25, or 42.52%, closing at $1.69 per share on July 10, 2025 [2] Group 2 - The Pomerantz Firm is recognized for its expertise in corporate, securities, and antitrust class litigation, having pioneered the field of securities class actions over 80 years ago [3] - The firm has a history of recovering multimillion-dollar damages for victims of securities fraud and corporate misconduct [3]

Core Viewpoint - HC Wainwright has initiated coverage on Ultragenyx Pharmaceuticals, Inc. with a Buy rating and a price target of $80, highlighting it as an attractive investment opportunity despite a 35% year-to-date decline in its stock price compared to a 2.7% decline in the XBI index [1][2]. Group 1: Company Performance and Trials - Ultragenyx's stock has faced pressure primarily due to investor disappointment regarding the interim results from the pivotal Orbit trial of UX143 (setrusumab) for osteogenesis imperfecta [2]. - The Phase 3 portion of the Orbit study is progressing towards a final analysis by the end of the year, with the second interim analysis not meeting the minimal p-value threshold to conclude the study early [3]. - The analyst believes the Orbit trial has not failed, and positive final results are anticipated, which should support a U.S. regulatory filing for setrusumab next year [4]. Group 2: Commercial Portfolio and Future Prospects - Ultragenyx's current commercial portfolio is estimated to support a fair value of approximately $35-$40 per share, excluding the potential contributions from its pipeline [4]. - The flagship Crysvita franchise is projected to achieve peak annual sales exceeding $3 billion [5]. - The company is expected to resubmit its Biologics License Application (BLA) for UX111 (ABO-102) later this year, with potential approval anticipated by mid-2026, addressing a high unmet need in the Sanfilippo syndrome market [6][7].

Core Viewpoint - Pomerantz LLP is investigating potential securities fraud or unlawful business practices involving Mereo BioPharma Group plc and its officers or directors [1] Group 1: Company Developments - On July 9, 2025, Mereo and Ultragenyx Pharmaceutical Inc. announced progress in the Phase 3 Orbit study for UX143 (setrusumab) in treating osteogenesis imperfecta, with a final analysis expected by the end of the year [3] - Following this announcement, Mereo's ADR price dropped by $1.25, or 42.52%, closing at $1.69 per share on July 10, 2025 [3] Group 2: Legal Context - Pomerantz LLP is recognized for its expertise in corporate, securities, and antitrust class litigation, having a long history of fighting for victims of securities fraud and corporate misconduct [4] - The firm has successfully recovered numerous multimillion-dollar damages for class members over its 80-year history [4]

Core Viewpoint - Pomerantz LLP is investigating potential securities fraud or unlawful business practices involving Ultragenyx Pharmaceutical Inc. and its officers or directors [1] Group 1: Company Developments - On July 9, 2025, Ultragenyx and Mereo BioPharma Group plc announced progress in the Phase 3 Orbit study for UX143 (setrusumab) in pediatric and young adult patients with osteogenesis imperfecta, with a final analysis expected around the end of the year [3] - Following the announcement, Ultragenyx's stock price dropped by $10.41 per share, or 25.11%, closing at $31.04 per share on July 10, 2025 [3] Group 2: Legal Context - The Pomerantz Firm is recognized for its expertise in corporate, securities, and antitrust class litigation, having a history of recovering multimillion-dollar damages for victims of securities fraud and corporate misconduct [4]

Group 1 - Pomerantz LLP is investigating claims on behalf of investors of Ultragenyx Pharmaceutical Inc. regarding potential securities fraud or unlawful business practices by the company and its officers [1] - On July 9, 2025, Ultragenyx and Mereo BioPharma Group announced the progress of the Phase 3 Orbit study for UX143 (setrusumab) in patients with osteogenesis imperfecta, with a final analysis expected by the end of the year [3] - Following the announcement, Ultragenyx's stock price dropped by $10.41, or 25.11%, closing at $31.04 per share on July 10, 2025 [3] Group 2 - The Pomerantz Firm is recognized for its expertise in corporate, securities, and antitrust class litigation, having pioneered the field of securities class actions over 80 years ago [4] - The firm has a history of recovering multimillion-dollar damages awards for victims of securities fraud and corporate misconduct [4]

Core Viewpoint - Ultragenyx Pharmaceuticals faces a regulatory setback as the FDA issued a Complete Response Letter (CRL) for its biologics license application (BLA) for UX111, a gene therapy for Sanfilippo syndrome type A (MPS IIIA) [1][6]. Regulatory Update - The CRL requested additional information and enhancements related to chemistry, manufacturing, and controls, stemming from recent facility inspections. The issues are facility- and process-related, not linked to product quality, and can be addressed quickly [2][6]. - Ultragenyx plans to collaborate closely with the FDA to resolve the observations and expects to resubmit the BLA, which will initiate a review period of up to six months [2]. Clinical Data and Impact - The FDA acknowledged the robustness of the neurodevelopmental outcome data and supportive biomarker findings. However, updated clinical data from current patients is required for the resubmission [3]. - The regulatory delay has pushed the potential approval of UX111 for MPS IIIA to 2026. Year-to-date, shares of Ultragenyx have decreased by 30.5%, contrasting with a 0.9% decline in the industry [3]. Disease Context - MPS IIIA is a rare, fatal lysosomal storage disease affecting the brain, with no approved treatments available. Approximately 3,000 to 5,000 patients are affected, with a median life expectancy of 15 years [9]. Pipeline Overview - Ultragenyx has several other gene therapy candidates in its pipeline, including UX143 for osteogenesis imperfecta and UX701 for Wilson disease, with ongoing studies showing promising results [11][12]. - The investigational gene therapy UX111 has received multiple designations in the U.S. and EU, including Regenerative Medicine Advanced Therapy and Orphan Drug designations [10].

Core Insights - Ultragenyx Pharmaceutical (RARE) and Mereo BioPharma (MREO) have provided an update on the mid-to-late-stage Orbit study for UX143 (setrusumab) aimed at treating osteogenesis imperfecta (OI) in pediatric and young adult patients, with final analysis expected by the end of 2025 [1][5] - The shares of both companies experienced a significant decline in premarket trading following the announcement of the delay in new data from the Orbit study [2][5] - UX143 is a fully human monoclonal antibody that inhibits sclerostin, and interim safety data has shown an acceptable safety profile, allowing the study to proceed as planned [2][5] Study Details - The Orbit study is assessing the impact of UX143 on fracture rates in patients aged 5 to 25 with OI, with a pivotal phase III portion enrolling 159 additional patients [7] - The primary efficacy endpoint of the Orbit study is to reduce the annualized clinical fracture rate, with participants moving into an open-label extension period after the primary analysis [7] - The Cosmic study targets a younger population aged two to under seven years, comparing UX143 to intravenous bisphosphonates for fracture reduction, with 69 patients enrolled [8] Partnership and Market Context - Ultragenyx Pharmaceuticals and Mereo BioPharma entered a licensing agreement in 2020 for the joint development of UX143, with RARE holding exclusive rights in several regions [9] - The partnership was expanded in 2024 through a manufacturing and supply agreement, with RARE responsible for supplying setrusumab to MREO [9] - OI affects approximately 60,000 individuals in commercially accessible geographies, and there are currently no approved treatments available [11]

Core Viewpoint - Ultragenyx Pharmaceutical Inc. and Mereo BioPharma Group plc stocks are experiencing significant declines following the update on the Phase 3 Orbit study for UX143 (setrusumab) in patients with osteogenesis imperfecta (OI) [1][2]. Group 1: Study Progress and Analysis - The Phase 3 portion of the Orbit study is progressing towards a final analysis expected by the end of the year, in line with the original plan [2]. - The second interim analysis did not meet the minimal p-value threshold of p<0.01, which would have allowed the study to conclude early [3]. - The Data Monitoring Committee confirmed that UX143 has an acceptable safety profile, recommending the continuation of the study [3][4]. Group 2: Future Expectations - The final analyses for the Orbit and Cosmic studies will occur after patients have been on therapy for at least 18 months, with thresholds set at p<0.04 for Orbit and p<0.05 for Cosmic [5]. - Analysts express optimism regarding the final analysis, anticipating a positive outcome due to longer follow-up duration and a lower statistical significance threshold [6]. Group 3: Market Reaction - Following the update, RARE stock has dropped by 26.2% to $30.60, while MREO stock has decreased by 34.7% to $1.919 [7].